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CHMP backs orphan drugs Uptravi and Coagadex

EMA advisors set Actelion and Bio Products therapies on path to European approval


At its meeting last week, the EMA's Committee for Medicinal Products for Human Use (CHMP) recommended approval of three orphan drugs for rare disorders.

The panel backed Coagudex (factor X concentrate) from Bio Products Laboratory for the treatment of factor X deficiency, a rare inherited bleeding disorder, along with Actelion's Uptravi (selexipag) for pulmonary arterial hypertension (PAH).

It also gave a green light to Bristol-Myers Squibb and AbbVie's multiple myeloma therapy Empliciti (elotuzumab), the first immuno-oncology therapy for the haematological cancer (for more details on this see our article here).

Like Empliciti, Coagudex is being reviewed under the EMA's accelerated assessment procedure and has been recommended for approval for the treatment, prevention and perioperative management of patients with factor X deficiency. The product works as replacement therapy and temporarily increases plasma levels of factor X, helping to prevent and control bleeding, according to the EMA.

Uptravi has been backed for the treatment of adults with PAH functional classes II-III - in other words patients who have slight to marked limitations in physical activity but who are generally comfortable at rest. PAH refers to high blood pressure that occurs in the arteries that connect the heart to the lungs and can cause serious complications including heart failure and arrhythmia.

Uptravi was first approved by the US FDA last December and went on sale the following month with an annual price tag of $160,000-$170,000. It has been tipped to become a $1bn-plus product for Actelion.

A CHMP recommendation is routinely followed within three months by a full European approval from the European Commission, so should see Uptravi, Coagadex and Empliciti licensed in the region by May.

The CHMP also gave positive opinions for three generic medicines developed by Mylan - amlodipine/valsartan for high blood pressure, rasagiline for Parkinson's disease and zonisamide for epilepsy, as well as new indications for Celgene's multiple myeloma therapy Revlimid (lenalidomide) and Novartis' Revolade (eltrombopag) for idiopathic thrombocytopenic purpura (ITP).

Revlimid has been backed for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL), while use of Revolade can now be used in children with ITP aged over one, in addition to adults.

The CHMP has also started a review of medicines containing the diabetes drug metformin after the Netherlands regulator raised a question about varying advice on its use in patients with reduced kidney function.

The meeting also saw the adoption of a final scientific opinion on experimental Ebola therapies, which is due to be published on the EMA website shortly.

Article by
Phil Taylor

5th February 2016

From: Regulatory



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